A groundbreaking clinical trial is showing early promise for adult stem cell transplants in treating advanced dry age-related macular degeneration (AMD), one of the leading causes of permanent vision loss in adults over 60 in the United States. The initial results suggest the treatment is safe and can significantly improve vision—even in patients with severe vision impairment.
The trial, published in Cell Stem Cell, involved transplanting retinal pigment epithelial stem cells sourced from adult postmortem eye tissue into the eyes of individuals with advanced dry AMD. The study’s first phase focused on evaluating safety and involved six participants who received a low dose of 50,000 cells during eye surgery. All patients tolerated the treatment well, with no serious inflammation or tumor growth reported.
Remarkably, these patients experienced measurable vision gains in their treated eyes, while the untreated eyes did not show comparable improvement. Participants in the low-dose group were able to read an average of 21 additional letters on a standard eye chart one year after receiving the stem cell therapy.
Dr. Rajesh C. Rao, the study’s lead author and Leonard G. Miller Professor of Ophthalmology & Visual Sciences at the University of Michigan, expressed excitement over the extent of vision improvement, particularly in those most severely affected by AMD. According to Dr. Rao, “[t]his level of vision gain has not been seen in this group of patients with advanced dry AMD.”
Researchers are now monitoring 12 additional participants who have received higher doses of 150,000 and 250,000 cells. If these higher doses continue to show safety, the study will advance to later clinical trial stages.
AMD affects about 20 million adults in the U.S., primarily impacting the central region of the retina (the macula) and resulting in loss of sharp, central vision. Currently, no treatment exists to restore vision lost to advanced dry AMD, making these findings an important step toward future regenerative therapies.
